Ultragenyx Pharmaceutical RARE announced that the FDA has granted the Breakthrough Therapy designation to its investigational candidate, setrusumab (UX143), as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients aged two years and older.
Setrusumab, a fully human monoclonal antibody, is an inhibitor of the sclerostin protein, which is responsible for hampering the maturation and activity of bone-forming cells. Ultragenyx, in partnership with Mereo BioPharma, is currently evaluating the candidate in two late-stage studies, Orbit and Cosmic, for the OI indication in adult and pediatric patients, respectively.
OI is a group of genetic disorders that impact bone metabolism. It is characterized by increased bone fragility and a high risk of fractures. The condition affects approximately 60,000 people in the developed world. Currently, there are no approved treatments for this debilitating condition, representing a highly unmet medical need.
Benefits of the FDA’s Breakthrough Therapy Designation Grant
The FDA’s Breakthrough Therapy designation is a process that speeds up the development and review of drugs for serious or life-threatening conditions. This designation is granted when early clinical evidence suggests the drug may significantly improve over existing treatments on an important clinical measure. Drugs with this designation receive more intensive guidance and organizational support from senior FDA managers.
Year to date, shares of Ultragenyx have gained 9.6% against the industry’s 2.8% decline.
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The FDA's decision is supported by preliminary clinical data, which includes encouraging 14-month results from the phase II portion of the Orbit study, showing a rapid and significant reduction in fracture rates among patients upon treatment with setrusumab, as well as findings from the completed phase IIb ASTEROID study of the candidate, which was conducted by Mereo.
Per Ultragenyx, the FDA’s Breakthrough Therapy designation for setrusumab highlights the severity of OI and its impact on patients and their families. Management also stated that this designation not only acknowledges the significant clinical benefits observed in the phase II portion of the Orbit study but also supports efforts to swiftly deliver this investigational treatment to patients who currently lack approved options.
Setrusumab was added to RARE’s pipeline after the 2020 collaboration agreement with Mereo. It also currently enjoys the Orphan Drug designation in both the United States and EU, Rare Pediatric Disease designation in the United States and has been accepted into the European Medicine Agency's Priority Medicines program, for the OI indication.
Other Key Clinical Programs in RARE’s Pipeline
Apart from setrusumab, Ultragenyx has several interesting gene therapy candidates in its pipeline. The company is evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease.
In May 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged eight years and older achieved its primary endpoint with statistical significance. The GlucoGene study also met its key secondary endpoints. The next step for the company involves discussing these results with regulatory authorities to support a marketing application in 2025.
Ultragenyx is also planning to seek accelerated approval for its AAV gene therapy candidate, UX111, which is being developed for the treatment of Sanfilippo syndrome type A. A biologics license application for the same will likely be filed with the FDA in late 2024 or early next year.
The company is developing GTX-102 for treating patients with Angelman syndrome (AS). It is currently gearing up to initiate a phase III AS study, named Aspire, for GTX-102 by the end of 2024. RARE also anticipates initiating an open-label clinical study named Aurora to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals ANIP, Catalyst Pharmaceuticals CPRX and Alnylam Pharmaceuticals ALNY, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share (EPS) have moved up from $4.53 to $4.81. EPS estimates for 2025 have improved from $5.38 to $5.86, during the same period. Year to date, shares of ANIP have gained 1.3%.
ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Catalyst’s 2024 EPS have increased from $1.75 to $1.92. The consensus estimate for 2025 EPS has improved from $2.09 to $2.26, during the same period. Year to date, shares of CPRX have soared 17.4%.
Catalyst’s earnings beat estimates in three of the trailing four quarters while matching on the remaining occasion, the average surprise being 13.88%.
In the past 60 days, estimates for Alnylam’s 2024 loss per share have narrowed from $1.20 to 63 cents. Loss per shares estimates for 2025 have narrowed from 34 cents to 27 cents, during the same period. Year to date, shares of ALNY have risen 39.4%.
Alnylam’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 108.53%.
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